Gene Therapy

• ‘Every day matters’: One B.C. family’s race to save their little boy

  His family noticed Clyde was stumbling and could no longer use the stairs he was diagnosed with metachromatic leukodystrophy, a genetic disease that attacks the nervous system.

  Canada

  Jul 9
  
• ‘Suffering’: B.C. family desperate to get gene therapy for son with rare condition

  Navpreet and Stalin Gill told Global News that they began to notice their now three-year-old son, Gurmoh, having mobility issues around age one.

  Health

  Apr 8
  
• Advertisement
• FDA approves 2 gene therapies for sickle cell disease

  The U.S. FDA on Friday approved two gene therapies for sickle cell disease from Vertex Pharmaceuticals and CRISPR Therapeutics, as well as from bluebird bio.

  Health

  Dec 8, 2023
• Advertisement
• 3 Alberta siblings with rare, terminal disorder finally have hope for a treatment

  A family from Alberta with three children who have been diagnosed with the rare and fatal Batten disease has been given a new kind of hope due to an older medication.

  Health

  Aug 10, 2023
• Groundbreaking gene therapy gives the gift of sight to Quebec child

  Doctors at the Maisonneuve Rosemont ophthamology clinic performed groundbreaking surgery on an 11-year-old boy, reversing a rare genetic condition that can cause blindness.

  Canada

  May 29, 2023
• Ontario family seeks answers, treatment for child’s rare genetic disease

  'Her birthday is in October. We don't know what's going to be going on then,' said Jonelle Vlogiannitis of her daughter Avery's degenerative disease.

  Health

  Aug 16, 2022
• ‘Uncharted territory’: Toronto family of child with rare disease raises $3M for therapy

  'This could change his life,' said Terry Pirovolakis of a gene therapy clinical trial to treat son Michael's ultra-rare condition, SPG50.

  Health

  Aug 15, 2022
• Nova Scotia celebrates research milestone for patients with Fabry disease

  Nova Scotia is home to some of the world's first patients to receive an experimental gene therapy for treating Fabry disease and the newly-published study results are promising.

  Health

  Feb 28, 2021
• Advertisement
• ‘He’s the only child living with CLN2 in Saskatchewan’: community rallies for 4-year-old with rare disease

  The community is rallying behind a four-year-old Regina boy who has been diagnosed with a rare disease, hoping to one day help fund a cure.

  Health

  Aug 28, 2019
• Advertisement
• First in-body CRISPR study set to start in U.S. to treat form of blindness

  The experimental treatment aims to supply kids and adults with a healthy version of the gene they lack, using a tool that cuts or “edits” DNA in a specific spot. It’s intended as a one-time treatment that permanently alters the person’s native DNA.

  Science

  Jul 25, 2019